Companies priced their initial public offerings (IPOs) in two hot therapeutic spaces, immunotherapy and biosimilars, proving that the window remains open – at least for the right candidates, if not at the hoped-for yield. But another firm withdrew its IPO, which could mean tighter times ahead.

Angling to strengthen its position in Hunter syndrome by way of a method past the blood-brain barrier, Shire plc – already an investor in Armagen Technologies Inc. – paid $15 million up front and added an equity investment, sweetening the deal to develop the enzyme replacement therapy AGT-182 for somatic as well as central nervous system (CNS) effects of Hunter syndrome.

With its lead cancer drug unpartnered and a new CEO yet to be chosen, Synta Pharmaceuticals Corp. is advancing the phase II/III AML LI-1 trial to test the chemosensitizing mettle of the heat-shock protein 90 inhibitor ganetespib when paired with low-dose cytarabine (Ara-C) in acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome patients who are not eligible for intensive chemotherapy.

The end of a two-part hearing on ways to speed clinical trials and include more diverse populations without introducing risk or mistakes included testimony by Alkermes plc CEO Richard Pops, who used his five minutes to propose a new framework for patients' help with trials that he said "would fundamentally transform the way we approach these [drug] development programs."

Investors didn't wait for the outcome of a potential FDA advisory committee showdown with its class competitor to flee Sarepta Therapeutics Inc., acting instead on 144-week phase IIb results – data that the company characterized as favorable – with exon-skipping eteplirsen for Duchenne muscular dystrophy (DMD).

Riding high on the gene therapy wave, Bluebird Bio Inc. will more than replace its spend on last month's acquisition of Pregenen Inc. by way of selling 3 million shares of stock at $34 each for net proceeds of about $95.6 million.

A pair of identical phase III trials hit their endpoint targets using sustained-exposure ciprofloxacin (branded Auripro, formerly OTO-201) in pediatric patients with bilateral middle ear effusion – fluid buildup – requiring tympanostomy tube placement, and Otonomy Inc. remains on track for a new drug application (NDA) filing in the first quarter of next year.

With another set of positive, top-line phase III results for its pain drug BEMA (bioerodible mucoadhesive) buprenorphine, this time for back pain in opioid-experienced patients, Biodelivery Sciences International Inc.'s (BDSI) partner Endo Pharmaceuticals Inc. plans a sit-down with the FDA later this month to talk about a new drug application (NDA).

Regado Biosciences Inc.'s double-punch anticoagulant took one on the chin from Wall Street, as investors learned about an unplanned review by the data safety monitoring board (DSMB) of the phase III trial testing the drug previously known as REG1 in patients undergoing percutaneous coronary intervention (PCI).

With fast track FDA status for its lead compound against fibrosis in Duchenne muscular dystrophy (DMD) and new financial support from the main disease association through a philanthropy program based on promising preliminary phase Ib/IIa data with oral HT-100 (delayed-release halofuginone), Akashi Therapeutics Inc. takes its place in the race for a new DMD therapy.